EA202190299A1 - CONNECTIONS AND METHODS FOR REDUCING EXPRESSION ATXN2 - Google Patents

CONNECTIONS AND METHODS FOR REDUCING EXPRESSION ATXN2

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Publication number
EA202190299A1
EA202190299A1 EA202190299A EA202190299A EA202190299A1 EA 202190299 A1 EA202190299 A1 EA 202190299A1 EA 202190299 A EA202190299 A EA 202190299A EA 202190299 A EA202190299 A EA 202190299A EA 202190299 A1 EA202190299 A1 EA 202190299A1
Authority
EA
Eurasian Patent Office
Prior art keywords
methods
atxn2
connections
reducing
reducing expression
Prior art date
Application number
EA202190299A
Other languages
Russian (ru)
Inventor
Сьюзан М. Фрайер
Приям Сингх
Фрэнк РИГО
Паймаан Джафар-Неджад
Холли Кордашевич
Original Assignee
Ионис Фармасьютикалз, Инк.
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Publication date
Application filed by Ионис Фармасьютикалз, Инк. filed Critical Ионис Фармасьютикалз, Инк.
Publication of EA202190299A1 publication Critical patent/EA202190299A1/en

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
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    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

Abstract

Предложены соединения, способы и фармацевтические композиции для снижения количества или активности РНК ATXN2 в клетке или организме животного и в некоторых случаях для снижения количества белка атаксин-2 в клетке или организме животного. Такие соединения, способы и фармацевтические композиции применимы для ослабления по меньшей мере одного симптома или признака нейродегенеративного заболевания. Такие симптомы и признаки включают атаксию, нейропатию и образование агрегатов. Такие нейродегенеративные заболевания включают спиноцеребеллярную атаксию 2 типа (SCA2), боковой амиотрофический склероз (БАС) и паркинсонизм.Compounds, methods and pharmaceutical compositions are provided for reducing the amount or activity of ATXN2 RNA in an animal cell or body and, in some cases, for reducing the amount of ataxin-2 protein in an animal cell or body. Such compounds, methods, and pharmaceutical compositions are useful for ameliorating at least one symptom or sign of a neurodegenerative disease. Such symptoms and signs include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include type 2 spinocerebellar ataxia (SCA2), amyotrophic lateral sclerosis (ALS), and parkinsonism.

EA202190299A 2018-07-25 2019-07-25 CONNECTIONS AND METHODS FOR REDUCING EXPRESSION ATXN2 EA202190299A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862703240P 2018-07-25 2018-07-25
PCT/US2019/043424 WO2020023737A1 (en) 2018-07-25 2019-07-25 Compounds and methods for reducing atxn2 expression

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EA202190299A1 true EA202190299A1 (en) 2021-06-11

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US (2) US11078486B2 (en)
EP (1) EP3826645A4 (en)
JP (2) JP7411632B2 (en)
KR (1) KR20210038589A (en)
CN (1) CN112423767B (en)
AR (1) AR115847A1 (en)
AU (1) AU2019310097A1 (en)
BR (1) BR112021000308A2 (en)
CA (1) CA3106986A1 (en)
CL (1) CL2021000159A1 (en)
CO (1) CO2021000196A2 (en)
CR (1) CR20210100A (en)
EA (1) EA202190299A1 (en)
IL (1) IL280123A (en)
JO (1) JOP20210018A1 (en)
MX (1) MX2021000922A (en)
PE (1) PE20211398A1 (en)
PH (1) PH12021500007A1 (en)
SG (1) SG11202100077PA (en)
TW (1) TW202020154A (en)
UY (1) UY38316A (en)
WO (1) WO2020023737A1 (en)
ZA (1) ZA202100108B (en)

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SG10201906520RA (en) * 2014-04-01 2019-09-27 Biogen Ma Inc Compositions for modulating sod-1 expression
US20200283800A1 (en) * 2017-10-23 2020-09-10 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative diseases
SG11202006526YA (en) 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
US11078486B2 (en) 2018-07-25 2021-08-03 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ATXN2 expression
WO2021159008A2 (en) * 2020-02-07 2021-08-12 Maze Therapeutics, Inc. Compositions and methods for treating neurodegenerative diseases
EP4162047A1 (en) * 2020-06-09 2023-04-12 Roche Innovation Center Copenhagen A/S Guanosine analogues for use in therapeutic polynucleotides
WO2023107893A2 (en) * 2021-12-06 2023-06-15 The Board Of Trustees Of The Leland Stanford Junior University Treatment of neuorodegenerative diseases through the inhibition of ataxin-2
WO2023239747A2 (en) * 2022-06-06 2023-12-14 University Of Utah Research Foundation Methods of modulating atxn2 expression
WO2023250368A2 (en) * 2022-06-24 2023-12-28 Eli Lilly And Company Atxn2 rna interference agents
WO2024056902A2 (en) * 2022-09-16 2024-03-21 Christopher Shaw Compositions and methods for treating neurological diseases

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